Category Archives: Human Genetics

NEW YORK, Nov. 18, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) onNovember 17, 2020issued a composition of matter patent, U.S. Patent No. 10,837,028,with claims directed to the AAV vector used in PR001, Prevails experimental gene therapy program for the treatment of Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings.

We are excited to make important progress this year with PR001, which is being evaluated in the Phase 1/2 PROPEL trial for patients with Parkinsons disease with GBA1 mutations and in the Phase 1/2 PROVIDE trial for patients with Type 2 Gaucher disease, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. We are advancing clinical development of PR001 to make a potentially transformative difference for these patients who currently have no approved treatment options.

The Company recently announced that patient dosing has continued in the Phase 1/2 PROPEL clinical trial of PR001 for PD-GBA patients, and it expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021. The Company expects to initiate enrollment of the Phase 1/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease patients in the fourth quarter of 2020 and currently anticipates it will provide the next update on PR001 biomarker and safety data for nGD in 2021.

The U.S. Food and Drug Administration has granted Fast Track designations for PR001 for the treatment of PD-GBA and nGD. In addition, the FDA granted PR001 Rare Pediatric Diseasedesignation for the treatment of nGD, and Orphan Drugdesignation for the treatment of patients with Gaucher disease.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease with GBA1mutations (PD-GBA) and neuronopathic Gaucher disease (nGD); PR006 for patients with frontotemporal dementia withGRNmutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr.Asa Abeliovichin 2017, through a collaborative effort withThe Silverstein Foundationfor Parkinsons with GBA and OrbiMed, and is headquartered inNewYork, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevails gene therapy candidates to make a transformative difference for patients with neurodegenerative diseases; the expected timing of reporting additional interim data on a subset of patients from the PROPEL trial; and the anticipated timing of enrollment of and the next update on data from the PROVIDE trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors sections of the Companys most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Lisa QuTen Bridge Communications LQu@tenbridgecommunications.com678-662-9166

Investor Contact:investors@prevailtherapeutics.com

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Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR001 - GlobeNewswire

AstraZeneca said on Monday its vaccine, developed along with the University of Oxford, could be around 90 per cent effective (File | Reuters)

In another piece of good news for the world in its fight against the Covid-19 pandemic, AstraZeneca said on Monday its vaccine, developed along with the University of Oxford, could be around 90 per cent effective under one dosing regimen -- a half dose, followed by a full dose about a month later, instead of two full doses one month apart.

India Today TV consulting editor Rajdeep Sardesai spoke to Professor Adrian Hill, Vaccinologist and Director, Jenner Institute, and Professor of Human Genetics at Oxford University, on what lies ahead in the war against Covid-19. Excerpts from an interview:

Q: Is it a day to smile?

A: Yes, we can. We had zero hospitalisation cases from the people who were vaccinated. The one dosing regimen -- a half dose, followed by a full dose about a month later, instead of two full doses one month apart -- allows more people to be vaccinated with a planned vaccine supply. I think its really good news that the world has three promising vaccine candidates at the moment (the two others being from Moderna and Pfizer).

Q: Is it correct to say the Oxford vaccine doesnt require a stringent cold chain regimen?

A: Yes, you need to store the vaccine in fridge temperatures, sometimes in deep-fridge temperatures. It can be distributed around the world using the usual immunisation distribution systems.

Q: What will be the cost of the vaccine?

A: In high-income countries, during the pandemic, it will be three dollars per vaccine. After the pandemic, the price will remain the same in low-income countries, but the manufacturers may look to make a profit in high-income countries.

Q: When will the vaccine be ready for emergency use (among health workers, etc)?

A: We will soon file for emergency use authorisation. We will work with our India partner, the Serum Institute of India, and the Drugs Controller General of India (DCGI) to see how quickly we can move. Early January will be the target. But mass vaccination will take more time. India is a big country but Serum is also the worlds biggest vaccine manufacturer.

Q: Vaccine development is a time-taking process. What explains this dramatic success?

A: Our students take 10 years. Were now into month 10. Its been different in many ways. Theres been help from funders, regulators, international agencies... We have also been allowed to overlap phases of clinical trials. About 270 people have worked night and day. Additionally, there were 19 trial sites in the UK alone. Brazil has vaccinated 10,000 people. Its going on in South Africa. The US has vaccinated 10,000 people. Kenya has started it. Serum is doing it in India.

Q: Are you already celebrating?

A: Were not yet at the finish line. But we have overcome the final fence. The next few weeks of paperwork are going to be really busy. We hope to have celebrations by the year-end.

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Top Oxford vaccinologist explains whats ahead in the war against Covid-19 - India Today

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today reported financial results and business highlights for the fourth quarter and full year fiscal 2020 ended September 30, 2020.

We ended our fiscal year with record revenue and orders against the backdrop of a global pandemic and significant uncertainty, said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. While we are proud of the new products we introduced to aid in the fight against COVID-19, which complemented our revenue, it was our core synthetic biology and next-generation sequencing (NGS) product lines that drove our overarching success.

We have aggressive plans for growth and expansion in fiscal 2021 and beyond, continuing to build our foundation for sustained success across synthetic biology, NGS, biopharma and DNA data storage.

FISCAL 2020 FINANCIAL RESULTS

FISCAL 2020 FOURTH QUARTER FINANCIAL RESULTS

"Over the last year, we delivered on revenue, orders, margin and product pipeline in a very challenging environment, commented Jim Thorburn, CFO of Twist. We have a strong balance sheet and momentum moving into fiscal 2021, and look forward to an exciting year ahead.

Fiscal Fourth Quarter 2020 and Recent Highlights

Fiscal 2021 Financial Guidance

The following statements are based on Twists current expectations for fiscal 2021. The following statements are forward-looking, and actual results could differ materially depending on market conditions and the factors set forth under Forward-Looking Statements below. Twist does not plan to update, nor does it undertake any obligation to update, this outlook in the future.

For the full fiscal year 2021, Twist provided the following financial guidance:

Fiscal 2021 First Quarter Financial Guidance

For the first quarter of fiscal 2021, Twist provided the following financial guidance:

COVID-19 Considerations

During the three months ended September 30, 2020, financial results of the Company were not significantly affected by the COVID-19 outbreak. However, the extent to which the COVID-19 outbreak affects Twists future financial results and operations is subject to a high degree of uncertainty and will depend on future developments, including the duration, spread and treatment of the outbreak domestically and abroad.

Conference Call Information

The company plans to hold a conference call and live audio webcast for analysts and investors today at 8:00 a.m. Eastern Time to discuss its financial results and provide an update on the companys business. The call can be accessed by dialing (866) 688-0947 (domestic) or (409) 217-8781 (international) and refer to the conference ID 2947139. A telephonic replay of the conference call will be available beginning approximately four hours after the call through November 30, 2020 and may be accessed by dialing (855) 859-2056 (domestic) or (404) 537-3406 (international). The replay conference ID is 2947139. The webcast replay will be available for two weeks.

Given the circumstances globally, it is recommended to dial-in at most 15 to 20 minutes prior to the call start to reduce waiting times. If a participant will be listen-only, they are encouraged to listen via the webcast on Twists investor page.

About Twist Bioscience Corporation

Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by writing DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research.

Follow us on Twitter | Facebook | LinkedIn | YouTube

Investor Relations Information

Twist uses the investor relations section on its website as a means of complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor Twist's investor relations website in addition to following Twist's press releases, SEC filings, and public conference calls and webcasts.

Legal Notice Regarding Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements under the headings Fiscal 2021 Financial Guidance and Fiscal 2021 First Quarter Financial Guidance, future growth and expansion plans and Twists other expectations regarding its future financial performance, the impact of the COVID-19 pandemic on Twists future financial performance, and Twists ability to address the challenges posed by the business and economic impacts of COVID-19 pandemic, diversification and revenue growth across all product categories, introduction of new products, the use of our products by the healthcare sectors for the potential detection and treatment of diseases, and expectations regarding newly announced partnerships. Such forward-looking statements involve known and unknown risks, uncertainties, and other important factors that may cause Twists actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the risks and uncertainties of the duration, extent and impact of the COVID-19 pandemic, including any reductions in demand for our products (or deferred or canceled orders) globally or in certain regions; the ability to attract new customers and retain and grow sales from existing customers; risks and uncertainties of rapidly changing technologies and extensive competition in synthetic biology could make the products Twist is developing obsolete or non-competitive; uncertainties of the retention of significant customers; supply chain and other disruptions caused by the COVID-19 pandemic or otherwise; risks of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Twists patents or proprietary rights; and the risk that Twists proprietary rights may be insufficient to protect its technologies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Twists business in general, see Twists risk factors set forth in Twists Annual Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 12, 2020 and subsequent filings with the SEC. Additional risk factors may be described in the Risk Factors section of Twists Annual Report on Form 10-K to be filed with the SEC on or about November 25, 2020. In addition, many of the foregoing risks and uncertainties are, and could be, exacerbated by the COVID-19 pandemic and any worsening of global or regional business and economic environment as a result. We cannot at this time predict the extent of the impact of the COVID-19 pandemic and any resulting business or economic impact, but it could have a material adverse effect on our business, financial condition, results of operations and cash flows. Any forward-looking statements contained in this press release speak only as of the date hereof, and Twist Bioscience specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Twist Bioscience Corporation

Condensed Consolidated Statements of Operations

(Unaudited)

(in thousands, except per share data)

Three months ended September 30,

Twelve months ended September 30,

2020

2019

2020

2019

32,432

15,736

90,100

54,385

17,578

12,386

61,406

47,426

11,636

10,496

43,006

35,683

27,185

24,423

103,267

80,126

-

-

22,500

-

56,399

47,305

230,179

163,235

(23,967

)

(31,569

)

(140,079

)

(108,850

)

112

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Twist Bioscience Reports Fourth Quarter and Full Year Fiscal 2020 Financial Results - Business Wire

A hub of deep expertise, the Department of Human Genetics helps partners across UCLA interpret data and leverage genomic technology to improve study design and solve medical problems.

We demystify genetic complexities to provide vital insights for a range of clinical and research applications. We strive to improve the care of as many patients as possible by pushing our capabilities, developing novel ways to address unanswered questions.

Your next collaboration is right down the street.

Our enviable proximity to the worlds brightest scientific minds enables both thriving scheduled events and impromptu sidewalk powwows. A casual conversation during your coffee run could lead to your next big publication.

Come find out why innovation lives here.

LEARN MORE

Steve Horvath, PhDThe precision of the epigenetic clock is the methodology that accurately measure biomarker of aging and could measure child development.Learn More

Chongyuan Luo, PhDThe Department of Human Genetics welcomes Dr. Chongyuan Luo as our new Assistant Professor to our team of world class faculty researchers here at the UCLA.Learn More

Sriram Sankararaman, PhDArun Durvasula

Science Advancespublished a paper on a study that uses genetic information to identify archaic 'ghost population'. PhD Candidate Arun Durvasula is first author and Principal Investigator Sriram Sankararaman serves as co-author. Both are from Human Genetics.Learn More

Yi Yin, PhD The Department of Human Genetics welcomes Dr. Yi Yin as our new Assistant Professor. She joined our team of world class faculty researchers in March 2020. Learn More

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Human Genetics - David Geffen School of Medicine at UCLA

SAN DIEGO, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) announced the publication of a study led by scientists and clinicians from the Institute for Human Genetics and the Benioff Childrens Hospital at the University of California, San Francisco (UCSF) that evaluated the ability of Bionanos optical genome mapping technology and another genome analysis method to diagnose children with genetic conditions who previously went undiagnosed by the standard of care methods alone. Of the 50 children in the study, the optical genome mapping results were sufficient to definitively diagnose 6 patients (or 12%) and, for another 10 patients (or 20%), the Bionano data revealed candidate pathogenic variants. Upon further analysis, it is expected that an additional 3 patients could be diagnosed with the Bionano data, bringing the total of definitively diagnosed patients to 9 (or 18%).

Erik Holmlin, Ph.D., CEO of Bionano Genomics commented, Increasing the number of patients who receive a definitive molecular diagnosis is the driving force behind much of the development of new diagnostic technologies. Every major change in medical guidelines connected to introducing novel methods has been driven by the ability of new methods to diagnose more patients than the previously existing standard of care. This study by the UCSF team shows that Bionanos optical genome mapping can potentially bring another such leap to the clinic by diagnosing many more patients than what existing chromosomal microarray (CMA) and whole exome sequencing (WES) can. Several studies released this year have shown that Saphyr can detect all clinically relevant variants identified by karyotyping, microarray and FISH in both leukemias and genetic disease cases. This UCSF study now shows in the largest cohort analyzed to date that Bionanos optical genome mapping diagnoses more patients than the traditional methods. We believe the increase in diagnosis over conventional methods can be a significant factor in Saphyr gaining widespread adoption as a clinical tool for genetic disease diagnosis and next-generation cytogenomics.

As described in the publication, the UCSF team performed full genome analysis by combining optical genome mapping with Bionano technology and linked-read sequencing on 50 undiagnosed patients with a variety of rare genetic diseases and their parents to determine if this full genome analysis method could help solve cases that had not been diagnosed with previous testing. Of the 50 cases, 42 were previously analyzed by CMA, the first tier medical test for genetic disease cases, and 23 had previously been analyzed with commercial trio whole exome sequencing, and no pathogenic or likely pathogenic variants were identified by these methods.

Bionanos optical genome mapping technology identified a number of pathogenic variants unidentified by CMA and undetectable by WES, including duplications and deletions that were too small to be identified by CMA, or occurred in regions of the genome not typically covered by CMA or WES. Of the additional 7 patients with variations considered to be candidates for pathogenic variants, the findings included deletions, duplications, and inversions. Before concluding that these variants are sufficient to diagnose the patients, further analysis is required since these variants had not previously been reported in patients with similar disease.

The publication is available at: https://www.medrxiv.org/content/10.1101/2020.10.22.20216531v1A recording of the webinar is available at: https://bionanogenomics.com/webinars/optical-mapping-in-rare-genetic-disease-diagnosis/

About Bionano GenomicsBionano is a genome analysis company providing tools and services based on its Saphyr system to scientists and clinicians conducting genetic research and patient testing, and providing diagnostic testing for those with autism spectrum disorder (ASD) and other neurodevelopmental disabilities through its Lineagen business. Bionanos Saphyr system is a platform for ultra-sensitive and ultra-specific structural variation detection that enables researchers and clinicians to accelerate the search for new diagnostics and therapeutic targets and to streamline the study of changes in chromosomes, which is known as cytogenetics. The Saphyr system is comprised of an instrument, chip consumables, reagents and a suite of data analysis tools, and genome analysis services to provide access to data generated by the Saphyr system for researchers who prefer not to adopt the Saphyr system in their labs. Lineagen has been providing genetic testing services to families and their healthcare providers for over nine years and has performed over 65,000 tests for those with neurodevelopmental concerns. For more information, visitwww.bionanogenomics.com or http://www.lineagen.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the contribution of Bionanos technology to the diagnosis of more genetic disease patients when compared to traditional standard of care methods; the capabilities of Bionanos technology in comparison to other genome analysis technologies; our expectations regarding the adoption of Saphyr as a clinical tool for genetic disease diagnosis and next-generation cytogenomics; and Bionanos strategic plans. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks and uncertainties associated with: the impact of the COVID-19 pandemic on our business and the global economy; general market conditions; changes in the competitive landscape and the introduction of competitive products; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of our technologies; the loss of key members of management and our commercial team; and the risks and uncertainties associated withour business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2019 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.

CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionanogenomics.com

Investor Relations Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 (617) 430-7577arr@lifesciadvisors.com

Media Contact:Darren Opland, PhDLifeSci Communications+1 (617) 733-7668darren@lifescicomms.com

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Largest Study To-Date Focused on Undiagnosed Genetic Disease Patients Reveals That Bionano's Optical Genome Mapping Technology Can Diagnose...

SAN FRANCISCO, Nov. 5, 2020 /PRNewswire/ -- Invitae Corporation (NYSE: NVTA), a leading medical genetics company, today announced financial and operating results for the third quarter ended September 30, 2020.

"Strategic, commercial and operating results in the third quarter continued to demonstrate the value and leverage available from our global, diversified business. These strong results are a testament to our unique combination of comprehensive menu, durable customer relationships and ability to execute," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "We further advanced clinical understanding of the importance of genetic information with the publication of several studies, including a collaboration supporting universal testing for cancer patients. Looking ahead, we believe recent acquisitions and integrations, coupled with our internal development efforts, will provide access to new and developing markets and improve ease-of-use for customers, enhancing our ability to meet the needs of patients and clinicians as the use of genetics in healthcare continues to accelerate."

Third Quarter 2020 Financial Results

Total operating expense, excluding cost of revenue, for the third quarter of 2020 was $102.9 million. Non-GAAP operating expense was $102.6 million in the third quarter of 2020.

Net loss for the third quarter of 2020 was $102.9 million, or $0.78 net loss per share, compared to a net loss of $78.7 million in the third quarter of 2019, or $0.82 net loss per share. Non-GAAP net loss was $81.7 million, or $0.62 non-GAAP net loss per share, in the third quarter of 2020.

At September 30, 2020, cash, cash equivalents, restricted cash and marketable securities totaled $368.0 million. Net decrease in cash, cash equivalents and restricted cash for the quarter was $61.4 million. Cash burn was $64.9 million for the quarter.

Corporate and Scientific Highlights

Webcast and Conference Call DetailsManagement will host a conference call and webcast today at 4:30 p.m. Eastern / 1:30 p.m. Pacific to discuss financial results and recent developments. To register for the conference call and webcast, please use one of the methods below. Upon registering, each participant will be provided with call details and a registrant ID.

Online registration: http://www.directeventreg.com/registration/event/7916067

Phone registration: (888) 869-1189 or (706) 643-5902

The live webcast of the call and slide deck may be accessedhere or by visiting the investors section of the company's website atir.invitae.com. A replay of the webcast and conference call will be available shortly after the conclusion of the call and will be archived on the company's website.

About Invitae

Invitae Corporation(NYSE: NVTA)is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

Safe Harbor StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the company's belief regarding the value and leverage of its global, diversified business; the company's belief regarding the impact of its unique combination of comprehensive menu, durable customer relationships and ability to execute; the importance of the company's recent studies and collaborations; the company's belief regarding the momentum of its business and ability to continue to deliver on its mission to bring genetic information into mainstream medicine; the impact of the company's acquisitions, including its completed merger with ArcherDX, as well as its internal development efforts, partnerships and product offerings; and the company's beliefs regarding its ability to access new and developing markets, improve ease-of-use for customers, and meet the needs of patients and clinicians. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the impact of COVID-19 on the company, and the effectiveness of the efforts it has taken or may take in the future in response thereto; the company's ability to continue to grow its business, including internationally; the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the risk that the company may not obtain or maintain sufficient levels of reimbursement for its tests; the company's failure to successfully integrate or fully realize the anticipated benefits of acquired businesses; risks associated with litigation; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Non-GAAP Financial MeasuresTo supplement Invitae's consolidated financial statements prepared in accordance with generally accepted accounting principles in the United States (GAAP), the company is providing several non-GAAP measures, including non-GAAP gross profit, non-GAAP cost of revenue, non-GAAP operating expense, including non-GAAP research and development, non-GAAP selling and marketing, non-GAAP general and administrative and non-GAAP other income (expense), net, as well as non-GAAP net loss and non-GAAP net loss per share and non-GAAP cash burn. These non-GAAP financial measures are not based on any standardized methodology prescribed by GAAP and are not necessarily comparable to similarly-titled measures presented by other companies. Management believes these non-GAAP financial measures are useful to investors in evaluating the company's ongoing operating results and trends.

Management is excluding from some or all of its non-GAAP operating results (1) amortization of acquired intangible assets, (2) acquisition-related stock-based compensation, (3) post-combination expense related to the acceleration of equity grants or bonus payments in connection with the company's business combinations, (4) adjustments to the fair value of acquisition-related liabilities and (5) acquisition-related income tax benefits. These non-GAAP financial measures are limited in value because they exclude certain items that may have a material impact on the reported financial results. Management accounts for this limitation by analyzing results on a GAAP basis as well as a non-GAAP basis and also by providing GAAP measures in the company's public disclosures.

Cash burn excludes (1) changes in marketable securities, (2) cash received from equity financings and (3) cash received from exercises of warrants. Management believes cash burn is a liquidity measure that provides useful information to management and investors about the amount of cash consumed by the operations of the business. A limitation of using this non-GAAP measure is that cash burn does not represent the total change in cash, cash equivalents, and restricted cash for the period because it excludes cash provided by or used for other operating, investing or financing activities. Management accounts for this limitation by providing information about the company's operating, investing and financing activities in the statements of cash flows in the consolidated financial statements in the company's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and by presenting net cash provided by (used in) operating, investing and financing activities as well as the net increase or decrease in cash, cash equivalents and restricted cash in its reconciliation of cash burn.

In addition, other companies, including companies in the same industry, may not use the same non-GAAP measures or may calculate these metrics in a different manner than management or may use other financial measures to evaluate their performance, all of which could reduce the usefulness of these non-GAAP measures as comparative measures. Because of these limitations, the company's non-GAAP financial measures should not be considered in isolation from, or as a substitute for, financial information prepared in accordance with GAAP. Investors are encouraged to review the non-GAAP reconciliations provided in the tables below.

INVITAE CORPORATION

Consolidated Balance Sheets

(in thousands)

(unaudited)

September 30,2020

December 31,2019

Assets

Current assets:

Cash and cash equivalents

$

106,436

$

151,389

Marketable securities

254,848

240,436

Accounts receivable

27,328

32,541

Prepaid expenses and other current assets

26,492

18,032

Total current assets

415,104

442,398

Property and equipment, net

46,130

37,747

Operating lease assets

39,007

36,640

Restricted cash

6,685

6,183

Intangible assets, net

187,060

125,175

Goodwill

211,225

126,777

Other assets

7,961

6,681

Total assets

$

913,172

$

781,601

Liabilities and stockholders' equity

Current liabilities:

Accounts payable

$

15,589

$

10,321

Accrued liabilities

77,986

64,814

Operating lease obligations

6,628

4,870

Finance lease obligations

1,237

1,855

Total current liabilities

101,440

81,860

Operating lease obligations, net of current portion

42,363

42,191

Finance lease obligations, net of current portion

1,834

1,155

Convertible senior notes, net

279,870

268,755

Deferred tax liability

10,250

Other long-term liabilities

60,864

8,000

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Invitae Reports $68.7 Million in Revenue Driven by 170,000 Samples Accessioned in the Third Quarter of 2020 - PRNewswire

News Release

Thursday, November 5, 2020

National Institutes of Health researchers have discovered a gene in mice that controls the craving for fatty and sugary foods and the desire to exercise. The gene, Prkar2a, is highly expressed in the habenula, a tiny brain region involved in responses to pain, stress, anxiety, sleep and reward. The findings could inform future research to prevent obesity and its accompanying risks for cardiovascular disease and diabetes. The study was conducted by Edra London, Ph.D., a staff scientist in the section on endocrinology and genetics at NIHs Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and colleagues. It appears in JCI Insight.

Prkar2a contains the information needed to make two subunits molecular components of the enzyme protein kinase A. Enzymes speed up chemical reactions, either helping to combine smaller molecules into larger molecules, or to break down larger molecules into smaller ones. Protein kinase A is the central enzyme that speeds reactions inside cells in many species. In a previous study, the NICHD team found that despite being fed a high fat diet, mice lacking functioning copies of Prkar2a were less likely to become obese than wild type mice with normally functioning Prkar2a.

The researchers determined that Prkar2a-negative mice ate less high-fat food than their counterparts, not only when given unlimited access to the food, but also after a fast. Similarly, the Prkar2a negative mice also drank less of a sugar solution than the wild type mice. The Prkar2a-negative mice were also more inclined to exercise, running 2-3 times longer than wild type mice on a treadmill. Female Prkar2a-negative mice were less inclined to consume high fat foods than Prkar2-negative males, while Prkar2-negative males showed less preference for the sugar solution than Prkar2-negative females.

Edra London, Ph.D., staff scientist in the NICHD Section on Endocrinology and Genetics, is available for comment.

London, E et al. Loss of habenular Prkar2a reduces hedonic eating and increases exercise motivation. JCI Insight. 2020.

About the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): NICHD leads research and training to understand human development, improve reproductive health, enhance the lives of children and adolescents, and optimize abilities for all. For more information, visit https://www.nichd.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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NIH researchers identify gene in mice that controls food cravings, desire to exercise - National Institutes of Health

The neocortex enables us to speak, dream and think. In search of the causes underlying neocortex expansion, researchers at the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden, together with colleagues at the University Hospital Carl Gustav Carus Dresden, previously identified a number of molecular players. These players typically act cell-intrinsically in the so-called basal progenitors, the stem cells in the developing neocortex with a pivotal role in its expansion.

The researchers now report an additional, novel role of the happiness neurotransmitterserotoninwhich is known to function in thebrainto mediate satisfaction, self-confidence and optimismto act cell-extrinsically as a growth factor for basal progenitors in the developing human, but not mouse, neocortex. Due to this new function, placenta-derived serotonin likely contributed to the evolutionary expansion of the human neocortex.

Abnormal signaling of serotonin and a disturbed expression or mutation of its receptor HTR2A have been observed in various neurodevelopmental and psychiatric disorders, such as Down syndrome, attention deficit hyperactivity disorder and autism. Our findings may help explain how malfunctions of serotonin and its receptor during fetal brain development can lead to congenital disorders and may suggest novel approaches for therapeutic avenues, [said study supervisor Wieland Huttner.]

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Experiencing happiness likely contributed to the dramatic growth and complexity of the human brain - Genetic Literacy Project

Amongst the various forms of discriminations the world has seen, Apartheid, a system of institutionalised racial segregation practised in South Africa, was one of the worst. I went to medical school with several South African students and the stories of discrimination they told me about were horrifying. Thankfully Apartheid, which was based on white supremacy and lasted more than 40 years, ended in the early 1990s.

I am often asked by my Caucasian friends if I felt discriminated against in the UK because of my brownness. I reply that it may have been true in early 1970s. I remember waiting in line to be served a pint of beer, only to be ignored by the proprietor of the pub. When I had the good fortune of going with a white girl, there was never a problem getting served.

Of course things have changed in the UK, which is today a proudly multicultural country. Overt discrimination of the kind that existed earlier is no longer tolerated. Asian communities especially are a thriving group, eagerly courted by political parties. Today when I go to a pub in London, I find the barmaid waiving me in.

This brings us to the topic of todays column: the role of race in health outcomes. Differences in health status, life expectancy and other indicators in different racial and ethnic groups are well documented. But before we get to that, let me get a somewhat controversial subject out of the way: the dominance of black athletes.

Black athletes enjoy a huge over-representation at the highest level of many sports - from the 100 metres sprint through to the marathon, every single record is currently held by athletes of African origin.

Sir Roger Bannister, who ran the first sub-4-minute mile, said in a speech to the British Association of Advancement of Science in 1995 that heel-bone length, subcutaneous fat and differences in the length of the Achilles tendon may explain the advantage that Africans may have in running sports. A study published in the Journal of Applied Physiology reported that Africans display an enhanced resistance to fatigue while running on a treadmill. There are tens of such studies - many of them controversial - which are beyond the scope of one column.

Coming back to race and health outcomes: Diabetes, for example, is 60 per cent more common among African Americans than white Americans. Black Americans are 2.5 times more likely to suffer limb amputations and 5.5 times likely to suffer from kidney disease because of diabetes.

Blacks in America are more likely to die of asthma than whites and, despite lower tobacco exposure, Blacks are 50 per cent more likely to get lung cancer.

Black Americans suffer twice the risk of stroke than Whites; strokes kill four times more 35- to 54-year-old Blacks than white. Black men have a 40 per cent high cancer death rate, and women 20 per cent higher death rate, than their white counterparts.

The stats for the other major minority group in America - the hispanics - are similarly depressing.

Around 15 years ago, the US FDA controversially approved a drug called BiDil for a single racial-ethnic group - African Americans - for treatment of congestive heart failure. The scientific research leading to BiDils approval tested the drug only in African American populations.

Craig Venter, the famous American biotechnologist who produced the map of the human genome, said it was disturbing to see things categorised in terms of race.

Is race really a surrogate marker for describing human genetic variation? In India genetics and race are certainly considered interchangeable. People still prefer getting married to someone of their own community. One of the major consequence of endogamy - the practice of marrying within the same community - is genetic diseases arising out of a limited gene pool. We would certainly be better off if we looked to widen our gene pool.

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Race, genetics, and their impact on health - Mumbai Mirror

THOUSAND OAKS, Calif., Nov. 4, 2020 /PRNewswire/ --Amgen (NASDAQ:AMGN) will present at the 29th Annual Credit Suisse Virtual Healthcare Conference at 4:15 p.m. ET on Monday, Nov. 9, 2020, in Scottsdale, Ariz. Murdo Gordon, executive vice president of Global Commercial Operations at Amgen and Peter H. Griffith, executive vice president and chief financial officer, will present at the conference. Live audio of the presentation can be accessed from the Events Calendar on Amgen's website, http://www.amgen.com, under Investors. A replay of the webcast will also be available on Amgen's website for at least 90 days following the event.

About AmgenAmgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to beone ofthe world'sleadingindependent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

For more information, visitwww.amgen.comand follow us onwww.twitter.com/amgen.

CONTACT: Amgen, Thousand OaksMegan Fox, 805-447-1423 (media)Trish Rowland, 805-447-5631(media)Arvind Sood, 805-447-1060 (investors)

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Amgen To Present At The 29th Annual Credit Suisse Healthcare Conference - WFMZ Allentown